A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia

计划入组 18 人 · 首次发布于 2021 年 5 月 24 日

Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia

首次发布于 2019 年 1 月 22 日

A Prospective Study of Pediatric Participants up to 16 Years of Age With Methylmalonic Acidemia (MMA) Due to Mutations in the MMUT Gene

计划入组 30 人 · 首次发布于 2026 年 2 月 25 日

Human methylmalonyl-CoA mutase gene therapy (Parexel International)

Human methylmalonyl-CoA mutase gene therapy (AAV, methylmalonic acidaemia)

(R)-methylmalonyl-CoA

C₂₅H₄₀N₇O₁₉P₃S

MUTAH UNIVERSITY

Jordan

Cota

The Coca-Cola Company

United States

Genespire and SR-TIGET Demonstrate Durable Preclinical Efficacy of Liver-Directed Gene Therapy for Methylmalonic Acidemia

Genespire Secures $52 Million to Advance Gene Therapy for Methylmalonic Acidemia

NIH to Advance Gene Therapy Trial for Rare Metabolic Disorder After Biotech Pullout